Potent aminocyclitol glucocerebrosidase inhibitors are subnanomolar pharmacological chaperones for treating gaucher disease.

Potent aminocyclitol glucocerebrosidase inhibitors are subnanomolar pharmacological chaperones for treating gaucher disease.

Amino-myo-inositol derivatives have been found to be potent inhibitors of glucocerebrosidase (GCase), the β-glucosidase enzyme deficient in Gaucher disease (GD). When tested using lymphoblasts derived from patients with GD homozygous for N370S or L444P mutations, the compounds enhanced GCase activit...

Disgrifiad llawn

Manylion Llyfryddiaeth
Prif Awduron:	Trapero, A, González-Bulnes, P, Butters, T, Llebaria, A
Fformat:	Journal article
Iaith:	English
Cyhoeddwyd:	2012

Eitemau Tebyg

Polyhydroxylated bicyclic isoureas and guanidines are potent glucocerebrosidase inhibitors and nanomolar enzyme activity enhancers in Gaucher cells.
gan: Trapero, A, et al.
Cyhoeddwyd: (2011)

Chaperoning glucocerebrosidase: a therapeutic strategy for both Gaucher disease and Parkinsonism
gan: Benjamin McMahon, et al.
Cyhoeddwyd: (2016-01-01)

Rational design and synthesis of highly potent pharmacological chaperones for treatment of N370S mutant Gaucher disease.
gan: Wang, G, et al.
Cyhoeddwyd: (2009)

Synthesis of N-substituted ε-hexonolactams as pharmacological chaperones for the treatment of N370S mutant Gaucher disease.
gan: Wang, G, et al.
Cyhoeddwyd: (2012)

Progranulin Recruits HSP70 to β-Glucocerebrosidase and Is Therapeutic Against Gaucher Disease
gan: Jinlong Jian, et al.
Cyhoeddwyd: (2016-11-01)

Effect of macrophage-targeted glucocerebrosidase(alglucerase) on hypolipidaemia of type 1 Gaucher disease(GD)
gan: Mistry, P, et al.
Cyhoeddwyd: (1997)

Docking study and biological evaluation of pyrrolidine-based iminosugars as pharmacological chaperones for Gaucher disease
gan: Kato, A, et al.
Cyhoeddwyd: (2016)

Abstract P-43: Effect of Glucocerebrosidase Dysfunction on the Pool of Plasma Exosomes of Patients with Gaucher Disease
gan: Luiza A. Garaeva, et al.
Cyhoeddwyd: (2021-06-01)

N- and C-alkylation of seven-membered iminosugars generates potent glucocerebrosidase inhibitors and F508del-CFTR correctors.
gan: Désiré, J, et al.
Cyhoeddwyd: (2014)

Engineering synthetic and recombinant human lysosomal β-glucocerebrosidase for enzyme replacement therapy for Gaucher disease
gan: Figueiredo, Lílian L. S., et al.
Cyhoeddwyd: (2024)

Engineering synthetic and recombinant human lysosomal β-glucocerebrosidase for enzyme replacement therapy for Gaucher disease
gan: Lílian L. Souza Figueiredo, et al.
Cyhoeddwyd: (2024-10-01)

Gaucher disease.
gan: Butters, T
Cyhoeddwyd: (2007)

Concise and enantioselective synthesis of the aminocyclitol core of hygromycin A.
gan: Donohoe, T, et al.
Cyhoeddwyd: (2005)

Recent progress in the synthesis of six-membered aminocyclitols (2008-2017)
gan: William A. Donaldson
Cyhoeddwyd: (2018-05-01)

Therapeutic delivery of recombinant glucocerebrosidase enzyme-containing extracellular vesicles to human cells from Gaucher disease patients
gan: Keatdamrong Janpipatkul, et al.
Cyhoeddwyd: (2024-10-01)

High throughput screening for small molecule therapy for Gaucher disease using patient tissue as the source of mutant glucocerebrosidase.
gan: Ehud Goldin, et al.
Cyhoeddwyd: (2012-01-01)

in silico identification of genetic variants in glucocerebrosidase (GBA) gene involved in Gaucher’s disease using multiple software tools.
gan: Madhumathi eManickam, et al.
Cyhoeddwyd: (2014-05-01)

Amphiphilic 1-deoxynojirimycin derivatives through click strategies for chemical chaperoning in N370S Gaucher cells.
gan: Diot, J, et al.
Cyhoeddwyd: (2011)

A new glucocerebrosidase-deficient neuronal cell model provides a tool to probe pathophysiology and therapeutics for Gaucher disease
gan: Wendy Westbroek, et al.
Cyhoeddwyd: (2016-07-01)

A myo-inositol dehydrogenase involved in aminocyclitol biosynthesis of hygromycin A
gan: Michael O. Akintubosun, et al.
Cyhoeddwyd: (2024-03-01)

Second-Generation Pharmacological Chaperones: Beyond Inhibitors
gan: My Lan Tran, et al.
Cyhoeddwyd: (2020-07-01)

Hydrophobic Rose Bengal Derivatives Exhibit Submicromolar-to-Subnanomolar Activity against Enveloped Viruses
gan: Anna A. Rubekina, et al.
Cyhoeddwyd: (2022-11-01)

Activation and Purification of ß‐Glucocerebrosidase by Exploiting its Transporter LIMP‐2 – Implications for Novel Treatment Strategies in Gaucher's and Parkinson's Disease
gan: Jan Philipp Dobert, et al.
Cyhoeddwyd: (2024-07-01)

A NOVEL SYNTHETIC AND RECOMBINANT GLUCOCEREBROSIDASE FOR GAUCHER DISEASE: IN SILICO MOLECULAR EVOLUTION AND GENE THERAPY APPROACHES TO ENHANCE ENZYME ACTIVITY
gan: LLS Figueiredo, et al.
Cyhoeddwyd: (2023-10-01)

INVESTIGATIONS ON THE RESISTANCE OF COMMENSAL SWINE ESCHERICHIA COLI TO SOME AMINOGLYCOSIDES-AMINOCYCLITOLS
gan: Valentina Urumova, et al.
Cyhoeddwyd: (2016-03-01)

Design of a New α-1-C-Alkyl-DAB Derivative Acting as a Pharmacological Chaperone for β-Glucocerebrosidase Using Ligand Docking and Molecular Dynamics Simulation
gan: Izumi Nakagome, et al.
Cyhoeddwyd: (2018-10-01)

Different and unusual presentation of Gaucher’s disease with the same mutation in the glucocerebrosidase enzyme (F266L) in two patients: a case report
gan: Bijan Keikhaei, et al.
Cyhoeddwyd: (2024-11-01)

Crystal structures of complexes of N-butyl- and N-nonyl-deoxynojirimycin bound to acid beta-glucosidase: insights into the mechanism of chemical chaperone action in Gaucher disease.
gan: Brumshtein, B, et al.
Cyhoeddwyd: (2007)

Regioselectivity in the Ring Opening of Epoxides for the Synthesis of Aminocyclitols from D-(-)-Quinic Acid
gan: Shu-Yu Yang, et al.
Cyhoeddwyd: (2012-04-01)

Synthesis of a new class of aminocyclitol analogues with the conduramine D-2 configuration
gan: Latif Kelebekli, et al.
Cyhoeddwyd: (2010-02-01)

Lysosomal lipid alterations caused by glucocerebrosidase deficiency promote lysosomal dysfunction, chaperone-mediated-autophagy deficiency, and alpha-synuclein pathology
gan: Alba Navarro-Romero, et al.
Cyhoeddwyd: (2022-10-01)

Quantification in the subnanomolar range of phospholipids and neutral lipids by monodimensional thin-layer chromatography and image analysis
gan: J I Ruiz, et al.
Cyhoeddwyd: (1997-07-01)

Small Molecule Chaperones for the Treatment of Gaucher Disease and GBA1-Associated Parkinson Disease
gan: Tae-Un Han, et al.
Cyhoeddwyd: (2020-05-01)

Cellular and biochemical response to chaperone versus substrate reduction therapies in neuropathic Gaucher disease
gan: Margarita M. Ivanova, et al.
Cyhoeddwyd: (2021-01-01)

Parkinsonisms and Glucocerebrosidase Deficiency: A Comprehensive Review for Molecular and Cellular Mechanism of Glucocerebrosidase Deficiency
gan: Emilia M. Gatto, et al.
Cyhoeddwyd: (2019-02-01)

Unveiling the Role of Cholesterol in Subnanomolar Ouabain Rescue of Cortical Neurons from Calcium Overload Caused by Excitotoxic Insults
gan: Dmitry A. Sibarov, et al.
Cyhoeddwyd: (2023-08-01)

Structural determinants for subnanomolar inhibition of the secreted aspartic protease Sapp1p from Candida parapsilosis
gan: Jiří Dostál, et al.
Cyhoeddwyd: (2021-01-01)

Comparative Study of Erythrocyte Sedimentation Rate after Aminoglycoside and Aminocyclitol Treatment in Goats (Capra hircus)
gan: Toncho DINEV, et al.
Cyhoeddwyd: (2016-07-01)

Imino sugar therapy for type 1 Gaucher disease.
gan: Priestman, D, et al.
Cyhoeddwyd: (2000)

N,Zn-Doped Fluorescent Sensor Based on Carbon Dots for the Subnanomolar Detection of Soluble Cr(VI) Ions
gan: Enoch Kwasi Adotey, et al.
Cyhoeddwyd: (2023-02-01)